ABM-1310 Lands FDA Fast Track Designation for BRAF V600E+ Glioblastoma

• Granting of a fast track designation (FTD) for ABM-1310 comes 2 months after the FDA granted an orphan drug designation.¹

• FTDs are designed to get promising therapies to patients sooner.

• Developer of ABM-1310, ABM Therapeutics, plans to work closely with the FDA to expedite the development of the drug.

The FDA has granted FTD to ABM-1310 for the treatment of patients with BRAF V600E-mutated glioblastoma multiforme (GBM).¹

ABM-1310 is an investigational small molecule BRAF inhibitor that is being development for the treatment of various BRAF V600E-mutant solid tumors. According to preclinical data, ABM-130 is highly water soluble, has cell permeability, and can penetrate the blood-brain barrier.²

“We are very grateful to the FDA for recognizing the potential of our novel next-generation investigational drug ABM-1310 to help patients with brain tumors,” said Zane Yang, MD, chief medical officer of ABM Therapeutics, in a press release.¹ “This offers ABM an interactive collaboration with the FDA to ensure ABM-1310 clinical development expeditiously with the highest standards of safety and quality.”

ABM-1310 is being studied in a phase 1, first-in-human, open-label, dose-escalation, and dose-expansion clinical trial (NCT04190628). The study will follow and 3 + 3 design by which patients in part A will receive ABM-1320 25 mg twice daily during a 28-day cycle. Patients in part B will receive the maximum-tolerated dose of the agent, as demonstrated in part A, and ABM-1310 will be combined with cobimetinib (Cotellic) for the first 21 days of the 28-day cycles. In part C, patients will be treated with the recommended phase 2 dose of ABM-1310, as determined in part A, alone or in combination with cobimetinib 60 mg administered the first 21 days of a 28-day cycle.²

The coprimary end points of the study include determining the maximum tolerated dose and recommended phase 2 dose of ABM-1310 in part A, the number of patients with treatment-related adverse events in part C, and the number of patients with abnormal laboratory values, in part C. the secondary end points of the study include the number of patients with treatment-related adverse events, the number of patients with laboratory abnormalities, pharmacokinetics, objective response rate, disease control rate, duration of response, progression-free survival, overall survival, and time to response.

The exploratory outcome measures being assessed in the study include preliminary efficacy in patients with certain types of BRAF V600 mutation, and the identification of major metabolite of ABM-1310 in patients who are treated with ABM-1310 monotherapy.

A total of 112 patients with either melanoma, GBM, colorectal cancer, cholangiocarcinoma, non–small cell lung cancer, thyroid cancer, or ovarian cancer will be enrolled in the trial. Patients are required to be 18 years of age or older with histologically or cytologically confirmed disease, be positive for a BRAF V600 mutation, a measurable lesion per RECIST v1.1, an ECOG performance status of 0 or 1 or Karnofsky performance status ≥ 70, and adequate organ function. Female patients must have a negative pregnancy test at screening, and all patients are required to use contraception during the study.

Patients who meet these criteria are being recruited at study sites in California, Florida, Michigan, New York, and Texas.

_REFERENCES:

_{1. ABM Therapeutics’ ABM-1310 Granted fast track designation by the FDA following orphan drug designation. News release. ABM Therapeutics, Inc. September 26, 2023. Accessed September 26, 2023. https://tinyurl.com/49embj6t}

_{2. Safety and tolerability of ABM-1310 in patients with advanced solid tumors. ClinicalTrials.gov. Updated August 29, 2023. Accessed September 26, 2023. https://tinyurl.com/mry7rvwb}